The mission of Columbus Children’s Foundation is driven by the urgency to accelerate equitable access to the most effective gene therapy solutions for children with ultra-rare genetic diseases.
Together we can change lives and ensure precious moments
are never lost.
Accelerating access to gene therapy for ultra-rare genetic disorders
The Columbus Children’s Foundation is driven by the urgency to advance gene therapy research to bring hope to children with ultra-rare genetic diseases.
Improving lives of children with ultra-rare diseases
Our goal is to see children going from wheelchairs to walking; from no speech to speaking, from lacking sight to seeing smiles; and from only receiving hugs to giving hugs.
Global Partner: Interventional NeuroTherapy Center, Brodno Hospital, Warsaw, Poland
Brodno Hospital provides avenues for advanced gene therapy treatments for the world’s rarest children. This is the first Interventional Neurology Center (INC) to open in Europe and the second in the world. Led by Krys Bankiewicz, doctors at the INC perform extremely precise operations to treat and cure children from around the world.
Marta M., Spain
Marta M. was just two months old when she was diagnosed as only the 100th child in Spain with Niemann Pick C disorder, an incurable neurodegenerative hereditary disease with a life expectancy of at best 20 years. Thanks to the support of the Fundacion Columbus, our European counterpart, Marta now has a life filled with hope.
The power of music
“We build bridges to help children born with ultra-rare diseases through the power of music. Just as a musical composition is a complex combination of perfectly tuned notes, so is the human genome. When in perfect harmony, both have amazing healing powers. Our worldwide concerts build awareness for children with ultra-rare diseases to raise needed funds.”
– Javier Garcia, CCF Board Trustee
Our latest news
Cell & Gene Therapy Insights: Curing ultra-rare diseases while ensuring access, equity and affordability
Yet again, it has been made possible – this time with the help of two donors. They would like to remain anonymous as what they are really interested in is being able to contribute to access to gene therapy for children with a rare neurodegenerative disease. Ted is from Swindon, UK. Like Irai, he suffers…
Marta is a four-and-a-half-year-old girl with Niemann Pick C. This is a rare neurodegenerative genetic disease that she was diagnosed with in its most severe form: that of childhood early onset. Despite the hardships of her daily life, Marta has decided to embark on a voyage to find a cure, with the hope and support…